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Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues.
Aartsma-Rus, Annemieke; Straub, Volker; Hemmings, Robert; Haas, Manuel; Schlosser-Weber, Gabriele; Stoyanova-Beninska, Violeta; Mercuri, Eugenio; Muntoni, Francesco; Sepodes, Bruno; Vroom, Elizabeth; Balabanov, Pavel.
  • Aartsma-Rus A; 1 Department of Human Genetics, Leiden University Medical Center , Leiden, the Netherlands .
  • Straub V; 2 John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University , Newcastle upon Tyne, United Kingdom .
  • Hemmings R; 2 John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University , Newcastle upon Tyne, United Kingdom .
  • Haas M; 3 Medicines and Healthcare Product Regulatory Agency , London, United Kingdom .
  • Schlosser-Weber G; 4 Central Nervous System and Ophthalmology, Scientific and Regulatory Management Department, Human Medicines Evaluation Division, European Medicines Agency , London, United Kingdom .
  • Stoyanova-Beninska V; 5 Bundesinstitut für Arzneimittel und Medizinprodukte , Bonn, Germany .
  • Mercuri E; 6 Medicines Evaluation Board , Utrecht, the Netherlands .
  • Muntoni F; 7 Department of Pediatric Neurology, Catholic University , Rome, Italy .
  • Sepodes B; 8 Centro Clinico Nemo, Policlinico Gemelli , Rome, Italy .
  • Vroom E; 9 Dubowitz Neuromuscular Center, UCL Great Ormond Street Institute of Child Health , London, United Kingdom .
  • Balabanov P; 10 Faculdade de Farmácia, Universidade de Lisboa , Lisboa, Portugal .
Nucleic Acid Ther ; 27(5): 251-259, 2017 Oct.
Article en En | MEDLINE | ID: mdl-28796573
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Terapia Genética / Exones / Distrofina / Oligonucleótidos Antisentido / Aprobación de Drogas / Distrofia Muscular de Duchenne Límite: Animals / Humans País como asunto: America do norte Idioma: En Año: 2017 Tipo del documento: Article
Texto completo
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PubMed Links
Search on Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Terapia Genética / Exones / Distrofina / Oligonucleótidos Antisentido / Aprobación de Drogas / Distrofia Muscular de Duchenne Límite: Animals / Humans País como asunto: America do norte Idioma: En Año: 2017 Tipo del documento: Article